Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

NCTID	NCT00643747 (View at clinicaltrials.gov)
Description	The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65. (Show More)
Development Status	Inactive
Indication	Leber Congenital Amaurosis-Type 2
Disease Ontology Term	DOID:0110016
Compound Name	TgAAG76
Compound Description	rAAV2/2.hRPE65p.hREP65
Sponsor	University College, London
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	12 (ACTUAL)
Results Posted	Not Available

Target Gene/Variant	RPE65
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Delivery System	Viral transduction
Vector Type	AAV2
Dose 1	1E11 vg
Dose 2	1E12 vg

Eligible Age	5 Years - 30 Years
Standard Ages	Child, Adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * Clinical diagnosis of severe early-onset retinal dystrophy confirmed missense mutation(s) in RPE65 Exclusion Criteria: * Visual acuity in the study eye better than 6/36 Snellen * Hypertension * Diabetes mellitus * Tuberculosis * Renal impairment * Immunocompromise * Osteoporosis * Gastric ulceration * Severe affective disorder) * Pregnancy or lactation View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	1
Locations	United Kingdom

Has US IND
FDA Designations
Recent Updates	Vector was originally produced by Targeted Genetics Corporation, clinical trial showed a lack of sustained efficacy